A Phase 1/2, Open-label, Multi-national, Multiple-cohort, Dose-escalation Study to Evaluate the Safety, Tolerability, and Efficacy of HG004 Gene Therapy in Subjects With RPE65-associated Leber Congenital Amaurosis Type 2 (LCA2)

HuidaGene Therapeutics Co., Ltd.1 site in 1 country20 target enrollmentOctober 31, 2023

ConditionsLeber Congenital Amaurosis Inherited Retinal Diseases Caused by RPE65 Mutations

InterventionsHG004

DrugsHG004

Overview

Phase: Phase 1
Intervention: HG004
Conditions: Leber Congenital Amaurosis
Sponsor: HuidaGene Therapeutics Co., Ltd.
Enrollment: 20
Locations: 1
Primary Endpoint: Incidence of ocular and non-ocular adverse events
Status: Recruiting
Last Updated: 2 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutations in RPE65 gene.

Registry

clinicaltrials.gov

Start Date

October 31, 2023

End Date

December 2025

Last Updated

2 years ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

HuidaGene Therapeutics Co., Ltd.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Male or females between 6 and 50 years of age at the time of signing the informed consent form.
•Willing to adhere to protocol as evidenced by written informed consent or parental permission and subject assent.
•Clinical confirmed diagnosis of Leber congenital amaurosis (LCA) and molecular diagnosis of LCA due to RPE65 mutations.
•Ability to perform tests of visual and retinal function.
•Visual acuity of ≤ 20/80 or visual field less than 20 degrees in the eye to be injected.
•Acceptable hematology, clinical chemistry, and urine laboratory parameters.

Exclusion Criteria

•Pre-existing eye conditions that would preclude the planned surgery or interfere with interpretation of study endpoints or complications of surgery (e.g., glaucoma requiring upcoming surgery, corneal or significant lenticular opacities).
•Presence of epiretinal membrane by OCT.
•Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
•Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter ocular function.
•Prior ocular surgery within six months.
•Prior gene therapy or oligonucleotide therapy treatments.
•Any condition which leads the investigator to believe that the participant cannot comply with the protocol requirements or that may place the participant at an unacceptable risk for participation.

Arms & Interventions

HG004

Intervention: HG004

Outcomes

Primary Outcomes

Incidence of ocular and non-ocular adverse events

Time Frame: 52 weeks

Secondary Outcomes

Change from baseline in visual fields of full-field stimulus threshold test in log cd.s/m2.(52 weeks)
Change from baseline in Best Corrected Visual Acuity (BCVA) of letters based on the Early Treatment Diabetic Retionpathy Study (ETDRS) chart(52 weeks)